MALVERN, Pa.—Ocugen, Inc. (NASDAQ: OCGN), a biotechnology company focused on discovering, developing and commercializing novel gene and cell therapies and vaccines, announced that the Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to the company’s investigational product OCU400 for the treatment of retinitis pigmentosa associated with RHO mutations. Receiving RMAT designation offers sponsor companies all the benefits of the fast track and breakthrough therapy designation programs, including early interactions with the FDA.

Ocugen is working with the FDA to finalize the Phase 3 protocol necessary to advance the clinical development for OCU400 to support an application for marketing authorization.

“RMAT designation is a significant accomplishment for the OCU400 clinical development program, as it validates the potential for our game-changing gene therapy approach to fulfill an unmet medical need for people who are facing blindness due to RP,” said Arun Upadhyay, PhD, chief scientific officer and head of R&D at Ocugen. “The FDA’s decision also reinforces the sense of urgency to bring a therapeutic option to these patients.”

Current data support the gene-agnostic mechanism of action for OCU400, which suggests that it may be able to provide treatment benefit to a broader group of RP and Leber congenital amaurosis (LCA) patients. Ocugen intends to submit additional efficacy and safety data for OCU400 in RP and LCA patients to the FDA in the future to potentially expand this RMAT designation to broader RP and LCA patient populations.